Screening Kids for Lipid Disorders; Oncology Drugs for Medicare Recipients

TTHealthWatch is a weekly podcast from Texas Tech. In it, Elizabeth Tracey, director of electronic media for Johns Hopkins Medicine in Baltimore, and Rick Lange, MD, president of the Texas Tech University Health Sciences Center in El Paso, look at the top medical stories of the week.

This week’s topics include screening kids for lipid disorders, treatment of Alzheimer’s disease, Medicare recipients and oncology drugs, and vaccinating moms for group B strep.

Program notes:

0:40 New monoclonal for Alzheimer’s

1:40 Decline in pace of progression

2:40 Amyloid in blood vessels

3:41 More coming on amyloid

3:55 Screening for lipid disorders in kids

4:55 No direct evidence of benefits or harms

5:55 More likely to be helpful than not

6:51 Moms given a group B strep vaccine

7:52 Immunogenicity of a single dose

8:53 Only a phase II trial

9:15 Utilization management in Medicare part D oncology drugs

10:15 Many million beneficiaries

11:15 Prior authorization common

12:08 End

Transcript:

Elizabeth: Should we screen kids for lipid disorders?

Rick: Treatment of early symptomatic Alzheimer’s disease.

Elizabeth: What does it look like when somebody on Medicare tries to get an oncology drug?

Rick: And a vaccine to improve the outcome of pregnancy.

Elizabeth: That’s what we’re talking about this week on TTHealthWatch, your weekly look at the medical headlines from Texas Tech University Health Sciences Center in El Paso. I’m Elizabeth Tracey, a Baltimore-based medical journalist.

Rick: And I’m Rick Lange, president of Texas Tech University Health Sciences Center in El Paso, where I’m also dean of the Paul L. Foster School of Medicine.

Elizabeth: Rick, I’m going to toss it right to you first. Let’s turn to JAMA, this very exciting and interesting drug that appears to be extremely beneficial with regard to Alzheimer’s disease.

Rick: This is not the first treatment that’s been approved for Alzheimer. This is a treatment that is designed to address the amyloid deposition in the brains of people with Alzheimer’s disease. You’ll recall that back in June of 2021, the FDA approved a monoclonal antibody for the treatment of early Alzheimer’s disease. Unfortunately, this particular drug really had unclear evidence of clinical efficacy and there were some issues regarding how it was approved. Two years later, it’s rarely used.

This particular antibody that was addressed in a study called TRAILBLAZER-ALZ 2 study is an antibody that does appear to have some benefit. It’s named donanemab and this is an 18-month, double-blind, placebo-controlled trial of people that have mild-stage dementia due to Alzheimer’s. This particular antibody is given once every 4 weeks.

Donanemab showed an impressive ability to remove amyloid from the brain. There was less worsening of cognitive decline in people that received donanemab compared with those that received placebo in both the primary endpoint and in 23 of 24 different secondary endpoints as well. This slowed the progression of dementia by a quarter to half a year over a 76-month period. Importantly, about half of the participants that had low to medium amyloid who received donanemab were considered stable, as opposed to only about 30% of people that received placebo.

Elizabeth: Of course, this is really excellent news. I think everyone is very excited about this outcome because, of course, Aduhelm was not very persuasive with regard to the data and was also extremely expensive. Let’s talk about that. Is there anything, any intimation, of what they’re planning to charge for this thing?

Rick: This is also going to be expensive. It’s a little simpler to administer, but it does carry some risk associated with it. When they looked at their brain scans, there was some swelling or some edema in the brain. There were some people that have bleeding. These are people that have amyloid angiopathy — that is, the amyloid proteins actually invaded the blood vessel.

Furthermore, this is not going to be easy to roll out. You have to make sure that somebody has Alzheimer’s as opposed to some other dementia. It requires looking at markers, screening, MRI scans, and perhaps PET scans. Then you have to follow individuals as well, so it’s not just the cost of the drug.

Elizabeth: I would note that the New England Journal is also publishing another trial on another monoclonal antibody and to me, this says, “Gosh, everyone is getting on the bandwagon with regard to developing these things.” My suspicion is that’s going to drive the costs down.

Rick: It will drive the cost of the medications down, but the cost and time to administer are still going to be expensive. These drugs are very effective at removing amyloid, but only modestly effective at changing the course of the cognitive decline, which suggests that there are other things besides amyloid protein that are also responsible for the dementia.

Elizabeth: It’s evident that there are very many different subcategories, even among Alzheimer’s dementia.

Rick: Absolutely. There will be a lot more coming over the next several years regarding treatment of Alzheimer’s related to amyloid and other proteins as well.

Elizabeth: Let’s stay in JAMA and let’s take a look at the USPSTF’s recommendation on screening for lipid disorders in children and adolescents. They, as they always do, took a look at all the literature that was out there. They found almost half a million kids who were assessed; there were no randomized controlled trials that directly assessed screening effectiveness and harms among kids for these lipid disorders. If I go all the way to the end of the discussion of these recommendations, it just points to a need for research.

Overall, the prevalence of elevated total cholesterol was 7.1% to 9.4% among this cohort, and of any lipid abnormality, was almost 1 in 5 kids. I had no idea that it was that prevalent.

What do we do about this? We do have kids who have familial hypercholesterolemia, so we can start identifying that they might have a problem in childhood. They note that observational studies suggest that statin treatment for this starting in childhood or adolescents reduces long-term cardiovascular disease risk.

However, at the end of the day, they said there is no direct evidence on benefits or harms of pediatric lipid screening. They were not able to actually recommend this. I would say that the editorialist goes through quite a lot about this particular decision and ultimately says this leaves a void for clinicians who are seeking to provide care to their patients with regard to this issue.

Rick: What we’re most concerned about is a condition called familial hypercholesterolemia, which occurs in about once in every 250 to 350 children where they have markedly elevated levels of LDL cholesterol that starts at birth. If it’s left untreated, it increases the risk of morbidity and mortality from coronary heart disease in adulthood by about two- to fourfold. Those are the kids you want to try to target.

The editorialist mentions there is probably no harm in checking cholesterol at least once in a child before the age of 10. The real question is, we don’t really know whether the treatment will be helpful, although it’s more likely to be helpful than not. Secondly, is when do you initiate it? Should it be initiated before age 10, or between 10 and 20, or after the age of 20? As you mentioned, we need studies to assess it even if we identify it.

Elizabeth: Yeah. The other thing that the editorialist notes, of course, is that we do routinely screen for other things at birth, like phenylketonuria and, sure, other things that have fatal outcomes versus those that are going to be a problem later on. But adding some kind of screening to that mix seems like, at least for familial hypercholesterolemia, not a big lift.

Rick: The things that we screen for at birth are the things that cause problems really soon after birth. They prevent the development of a normal childhood. These high cholesterols associated with familial hypercholesterolemia occur over the course of decades. It may not be important that we screen at birth, but it’s probably important that we screen sometime before they finish their adolescent years.

Elizabeth: Let’s turn to the New England Journal talking about kids. Can we give moms a vaccine for Group B strep?

Rick: The reason why Group B strep is important — it’s a common cause of both sepsis and meningitis in newborns through the first 3 months of their life. Group B strep is oftentimes passed to the child during delivery. Frequently, since about 2005, if a mother has been screened — and they should be screened to determine whether they are infected with Group B strep — is they are given prophylactic antibiotics prior to delivery. This has actually decreased some of the complications associated with a group B strep infection during delivery, but it hasn’t affected the mortality up to 3 months afterwards.

This is a study that looked at the potential for administering a vaccine for Group B strep to see whether it would increase antibodies in the mother, would they be passed to the baby, and would the level of antibodies be sufficient to cause protection. This is a surrogate study. It’s a Phase II, placebo-controlled trial involving pregnant women looking at both the safety and the immunogenicity of a single dose of a vaccine targeted towards Group B strep.

Among over 1,700 participants in whom they examined the presence of antibodies and the risk of Group B strep infections among children, they determined the protective level of antibodies in mothers. Then they provided the vaccine in six formulations in three different dose groups. They looked at the maternal immune responses and determined that they were actually pretty robust. These vaccine antibodies were actually passively transferred to the infants at birth. What they determined was that they developed sufficient antibodies associated with about a 75% risk reduction at birth.

Elizabeth: Congratulations to these authors, of course, for doing a study in a population that’s extremely difficult to do studies in.

Rick: As you mentioned, doing studies in pregnant women requires a tour de force.

Elizabeth: Good news, we’re happy to hear that. How long do you think it’s going to be before it gets rolled out? This is only a Phase II.

Rick: It is Elizabeth. I suspect that this will be rolled out pretty quickly because there is nothing quite like it. This pathogen has caused about 46,000 stillbirths, 40,000 cases of invasive maternal disease, and about a quarter of a million cases of early onset or 162,000 cases of late onset disease in infants. Those that recover, about 37,000 have moderate or severe neurodevelopmental injury, so it’s a serious problem.

Elizabeth: Finally, let’s go back to JAMA and we’re going to take a look at a research letter that’s looking at utilization management trends in Medicare Part D oncology drugs. Of course, our oncology drugs, we mentioned recently, have been much in the news because there have been a lot of shortages of them. Sometimes those are shortages that result in significant clinical impact in terms of somebody’s management of their cancer.

In this case, they are looking at use of these drugs between 2010 and 2020. They basically posit that prior authorization and other utilization management techniques disproportionately affect oncology treatments. We know that oncology treatments are frequently in the Medicare population. So they look at this, as I said, in the Part D formulary files and look at prior authorization quantity limits and step therapy for each unique drug dose formulary combination of orally administered oncology drugs.

They found an unbelievable number — almost 30 million beneficiaries — enrolled in 333 formularies covering 62 oral oncology drugs. That was in 2010. In 2020, it was 47 million.

The proportion of drug dose formulary combinations that required prior authorization increased over time and that’s sort of not surprising. For specialty brand drugs, it increased from 73% to 95%. Quantity limits also increased from 31% to 63% about and specialty generics from 33% to 78% in this interval. What this study shows is that, gosh, it is clear that they are trying to really limit the utilization of these drugs.

Rick: Yeah. In fact, in their discussion they mentioned that the utilization management for these Medicare Part D oncology drugs not only increased, but prior authorization was the most prevalent strategy for specialty brand drugs as well as non-specialty brand drugs. That’s how they limit their use. That’s really disappointing, because it’s really not clear to me why prior authorization is really even required for what we know are being highly effective, first-line drugs and especially for ones that are even generic.

Elizabeth: Exactly. Now, two things to address on the oncology drugs utilization. One is this — prior authorization for Medicare beneficiaries — and the other, of course, is the shortages. We seem to be seeing a lot of this.

On that note then, that’s a look at this week’s medical headlines from Texas Tech. I’m Elizabeth Tracey.

Rick: And I’m Rick Lange. Y’all listen up and make healthy choices.

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